DRUG
migalastat HCl 150 mg
150 mg capsule
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
Status
Available
Phase
Not applicable
Enrollment
—
Locations
0
Results
Not posted
Publications
0
Study summary
What the protocol is testing.
This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific patients with Fabry disease. Treatment is open label.
Full detailed description
This Physician Initiated Request program allows physicians to request permission from Amicus to receive migalastat HCl for specific patients with Fabry disease who have a mutation amenable to this treatment, who do not have access to commercial Galafold or available treatment alternatives, or do not meet requirements for participation in an existing migalastat clinical study. Up to 20 patients worldwide may be treated. Patients must meet specific criteria to receive Amicus permission for participation. Key criteria for participation include: At least 2 years old; Confirmed GLA gene mutation shown to be responsive to migalastat; Have no treatment option because either unsuitable for enzyme replacement therapy (ERT) or unable to access ERT.
Interventions
Treatment arms and agents.
DRUG
migalastat HCl 20 mg
20 mg dispersible tablets; dosing will be based on body weight
Timeline
From registration to results.
First posted
Nov 22, 2011
Study start
Not reported
Primary completion
Not reported
Study completion
Not reported
Results posted
Not reported
Registry updated
Jul 3, 2025
Outcomes
What the study measures.
Primary outcomes
Not reported in the indexed record.
Secondary outcomes
Not reported in the indexed record.
Eligibility
Who can take part.
- Minimum age
- 2 Years
- Maximum age
- Not reported
- Sex
- ALL
- Healthy volunteers
- No
Inclusion Criteria: * Confirmed GLA mutation predicted to be responsive migalastat in the human embryonic kidney (HEK-293) cell-based assay * At least 2 years of age * Strong clinical indication for treatment of Fabry disease * No other treatment option including either unsuitable for ERT or unable to access ERT * Appropriate female and male contraception * Willing to receive treatment with migalastat HCl via this program including having signed an authorization for sharing clinical data Exclusion Criteria: * Scheduled for renal or other organ transplant or replacement therapy * Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy FABRAZYME® (agalsidase beta), REPLAGAL™ (agalsidase alpha), or Elfabrio® (pegunigalsidase alfa) * Contraindication to migalastat, i.e., sensitivity to other iminosugar such as miglustat, miglitol * Treated with another investigational drug within 30 days of start of migalastat HCl treatment * Unable to comply with study requirements or deemed otherwise unsuitable for study entry in the opinion of the investigator.
Study locations
0 registered sites.
No country data reported. Showing up to 24 locations stored in the fast local snapshot.
No study locations reported.
Publications
Results and literature.
No PMID-linked publications were present in this registry snapshot.
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Related PeptideStat pages
Put the record in context.
Research pages describe evidence. Vendor pages, where available, describe independently tracked research-product listings and are not clinical recommendations.